Severe Aplastic Anemia Clinical Trial
Official title:
To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.
Status | Recruiting |
Enrollment | 160 |
Est. completion date | December 31, 2025 |
Est. primary completion date | December 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 50 Years |
Eligibility | Inclusion Criteria: 1. Diagnosed as SAA/vSAA 2. Indication for hematopoietic stem cell transplantation 3. Available HLA matched sibling or unrelated donor 4. No active infection 5. No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency) 6. Signed informed consent 7. High risk factors of mixed chimerism, at least one of the following 1. Age < 18 years old 2. Ferritin level =2500ng/ml before transplantation Exclusion Criteria: 1. Age > 50 years old 2. ECOG=3 3. Active infections that were difficult to control 4. Severe liver and kidney dysfunction 5. Mental illness 6. Not signing the informed consent 7. pregnant or lactating women 8. Any condition considered by the investigators to be unsuitable for enrollment |
Country | Name | City | State |
---|---|---|---|
China | Deparment of Hematology, Peking University People's Hospital | Beijing | Beijing |
Lead Sponsor | Collaborator |
---|---|
Peking University People's Hospital |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Failure free survival | Failure free survival was defined as survival with a response to therapy. | 1 year post HSCT | |
Secondary | The incidence of mixed chimerism | The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells. | 1 year post HSCT | |
Secondary | Regimen related toxicity | The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988). | 100 days post HSCT | |
Secondary | Myeloid and platelet engraftment | Myeloid and platelet engraftment were defined as international criteria. | 100 days post HSCT | |
Secondary | The incidence of graft versus host disease | The severity of acute and chronic GVHD was evaluated according to standard criteria. | 100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD | |
Secondary | The incidence of CMV and EBV reactivation | The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia. | 100 days post HSCT | |
Secondary | The incidence of Transplantation related mortality | Transplantation related mortality was defined as death without disease progression. | 1 year post HSCT | |
Secondary | The probability of Overall survival | Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up | 1 year post HSCT |
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